Abstract
The MYCN oncogene is the single most powerful biologic prognostic factor yet identified in childhood neuroblastoma. Children whose tumours display MYCN amplification have a particular poor prognosis, and the possibility of delivering highly effective therapies that target the MYCN pathway is an area of research that is highly attractive. We have used a variety of approaches, to firstly identify and validate key genes associated with MYCN-driven neuroblastoma, and secondly to develop small molecule drugs to inhibit these genes.1,2 This has involved the use of cell-based assays and high throughput screening of chemical small molecule libraries. We have also employed chemical mutagenesis of neuroblastoma-prone transgenic mice as a powerful tool for elucidating new genes involved in neuroblastoma tumourigenesis. Our research is ultimately aimed at the development of new therapies for childhood neuroblastoma and our findings will be discussed.
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